Potential New Parkinson’s Treatment Passes Hurdle

A two-year long Phase 2 clinical trial of the nutritional supplement inosine has shown that the compound can safely and effectively raise levels of urate in the blood. This is an important step because scientists suspect that urate may slow the progression of Parkinson’s disease.

The results were published today in the journal JAMA Neurology. URMC neurologist Karl Kieburtz, M.D., M.P.H. – along with researchers at Massachusetts General Hospital and Harvard University – is one of the co-principal investigators of the study.

Urate is a naturally occurring compound found in blood. The first clue that higher than normal urate levels may provide relief to people with Parkinson’s disease dates back to the 1980s when a team of URMC researchers, including Kieburtz and Ira Shoulson, M.D., were analyzing results from a study called DATATOP. The study was one of the first of its kind in that it harnessed the collective efforts and resources of dozens of researchers across the country.

While the results of the specific treatment for Parkinson’s being studied under DATATOP were mixed, the data generated by thousands of blood, urine, and cerebral spinal fluid samples collected from the 800 plus study participants produced a treasure’s trove of information, including the observation that people with elevated urate levels seemed to be more protected from the effects of the disease.

Doctors currently have several drugs at their disposal to help patients manage the symptoms of Parkinson’s. However, over time these drugs tend to lose their effect. Consequently, one of the key objectives of current Parkinson’s research is to identify treatments that are “neuroprotective,” can slow the progression of the disease, and – combined with existing treatments – keep symptoms at bay for a longer period of time.

The focus of the current study – called SURE-PD – found that the drug could be administered in a dose that would safely increase urate levels without triggering the side effects commonly associated with high levels of the compound, such as kidney stones and gout.

The study was funded by the NIH and the Michael J. Fox Foundation. Read more about the results of the study here.

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